InterventionsAnimalsPreprint

In vivo HSPC gene therapy of hemoglobinopathies without drug selection of corrected cells

bioRxiv

The paper

Akshara Sakunthala Velmurugan, Kiriaki Paschoudi, Jack A. Queenan,
Show 17 more authorsSucheol Gil, Aidan Maynard, Charlie E Fontana, Reva Sharma, Anika Long, Chang Li, Hongjie Wang, Anna Kate Anderson, Aphrodite Georgakopoulou, Nikoletta Psatha, Maria Giannaki, Efthymia Vlachaki, Nouraiz Ahmed, Alexander A. Sousa, David R. Liu, Evangelia Yannaki, André Lieber,
Karthik V. Karuppusamy

University of Washington

bioRxiv · 17 Sep 2026 · Preprint, not peer-reviewed

doi.org/10.64898/2026.09.15.751289PubMed 42779985

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